The ability to exogenously control the expression of genes in mammalian cells has been a powerful tool of biomedical research. In particular, gene regulation technology has played a central role in efforts to understand the role of specific gene products in fundamental biological processes and in both normal development and disease states. It is likely that this form of genetic technology will continue to have impact in a variety of areas of basic research and may enable new therapeutic paradigms, such as the regulated delivery of protein therapeutics. The technology may also have significant future impact upon the safety of gene therapy strategies.
To date, most of the gene regulation systems commonly utilized are based on the control of transcription. Despite their considerable utility, these systems possess some significant limitations due to their reliance on chimeric transcriptional transactivators and specialized promoter elements. Such limitations include the requirement for co-introduction of genes encoding the relevant transcriptional transactivator along with the gene to be regulated, and the inability to provide for the “on-off” regulation of a gene in the context of its own endogenous transcriptional control elements.
Thus, there is a need for developing a novel gene regulation system that does not rely on the control of transcription.